Iminosugar-based inhibitors of glucosylceramide synthase increase brain glycosphingolipids and survival in a mouse model of Sandhoff disease.
The neuropathic glycosphingolipidoses are a subgroup of lysosomal storage disorders for which there are no effective therapies. A potential approach is substrate reduction therapy using inhibitors of glucosylceramide synthase (GCS) to decrease the synthesis of glucosylceramide and related glycosphin...
Päätekijät: | , , , , , , , , , , , , |
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Aineistotyyppi: | Artikkeli |
Kieli: | English |
Julkaistu: |
Public Library of Science (PLoS)
2011-01-01
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Sarja: | PLoS ONE |
Linkit: | https://www.ncbi.nlm.nih.gov/pmc/articles/pmid/21738789/pdf/?tool=EBI |